Kanuma (sebelipase alfa) is a hydrolytic lysosomal cholesteryl ester and triacylglycerol-specific enzyme.

Kanuma is specifically indicated for the treatment of patients with a diagnosis of Lysosomal Acid Lipase (LAL) deficiency.

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  • Kanuma
  • Adverse effects associated with the use of Kanuma may include, but are not limited to, the following:

    Patients with Rapidly Progressive Disease Presenting within the First 6 Months of Life (≥30%):

    diarrhea
    vomiting
    fever
    rhinitis
    anemia
    cough
    nasopharyngitis
    urticaria
    Pediatric and Adult Patients (≥8%):

    headache
    fever
    oropharyngeal pain
    nasopharyngitis
    asthenia
    constipation
    nausea
  • The recommended dose is as follows: Patients with Rapidly Progressive LAL Deficiency Presenting within the First 6 Months of Life: The recommended starting dosage is 1 mg/kg administered once weekly as an intravenous infusion. For patients who do not achieve an optimal clinical response, increase to 3 mg/kg once weekly. Pediatric and Adult Patients with LAL Deficiency: The recommended dosage is 1 mg/kg administered once every other week as an intravenous infusion.